the ability of cancer cells from one species (mice) to fight the growth of human tumors. Any profits from the venture are to be pumped back into Kluge’s separate charitable foundation. More recently, philanthropist John Flatley established a venture group to fund development of treatments for cystic fibrosis. Flatley, a real estate developer, started the new Flatley Discovery Lab because he felt that nonprofits were not the appropriate vehicle for funding ideas from entrepreneurs interested in developing treatments for CF. The most unusual feature of this venture fund? It’s stated goal is to break even, not return a profit to investors.
Open Source Opens a New Research Path (Funding Level: Millions)
The open source movement has flowed from software to scientific journals (e.g. PLoS, the Public Library of Science) and biomedical research (e.g. Sage Bionetworks). Much of the funding for the open source movement has come from philanthropic investments and government resources, but it also includes income from collaborative research partnerships as well as traditional grant support. Sage Bionetworks, for example, has just announced an oncology partnership with AstraZeneca. The question has been posed as to whether open source research and development can reinvigorate drug research. It has certainly stimulated thinking about novel ways to do drug discovery research. Transparency Life Sciences is in the process of being launched to do open-source drug development using telemedicine apps for clinical trial monitoring. Open source resources include the National Cancer Institute’s Biomedical Information Grid that allows cancer researchers to work together. A recent commentary pointed out the open source research will be facilitated by the creation of standards for different types of research data. This approach will enable a wider spectrum of users to access these large datasets. The Biobricks Foundation is a nonprofit group focused on coordinating the production of DNA “modules” encoding basic biological functions or structures. These modules can be combined in a synthetic biology approach towards engineering novel proteins. The P2P Foundation hosts some interesting discussions on the concept of open source biotechnology, with links to projects that both the public and private sector can tap in to.
When All Else Fails, Ask the Patients to Pay (Funding Level: Tens of Thousands)
Clinical trials are generally paid for by their sponsors, which are usually pharma or biotech companies (or sometimes the federal government). But what happens when a biotech company sponsoring a clinical trial runs out of money before the trials are completed? Suppose the company goes out of business, or can no longer afford to produce the drug being tested? This situation arose during a trial for ALS, which is classified as a rare disease because there are only about 20,000-30,000 patients in the U.S. The clinical trial ran short of money because the sponsoring company went out of business. As a result, patients were asked to pay for their drugs by the doctor who was running the trial. The burden of having to find funding for their own clinical trials may fall hardest on patients needing medicines being developed as orphan drugs (such as this ALS trial). Historically, few biotech companies have wanted to work on orphan drugs because these diseases have a smaller number of afflicted individuals. However, this trend has been reversed as pharma and biotech executives witnessed Genzyme’s commercial success in developing some of the worlds most expensive and profitable drugs for rare diseases. In 2010 alone the FDA granted 192 orphan drug designations out of 328 applications, illustrating the current strong interest in working on these rare diseases.
An interesting approach for developing new breast cancer treatments is being undertaken by the Pink Army Cooperative. This organization asks individuals to join their cooperative by contributing $20 to fund the development of oncolytic viruses that will be created (using open source synthetic biology technologies) for treating breast cancer. Members of the cooperative will gain access to any treatments that arise from this funding. Unfortunately, details are in short supply on the organization’s Website, and it is unclear how many co-op members have signed on since the group was launched in 2009.
Summary
This survey was not meant to be exhaustively comprehensive, but was intended to illustrate that a wide variety of funding sources are being used to pay for various stages of the drug development process. The decline in the number of new drug approvals over the past 10 years indicates that a variety of developmental approaches are both necessary and desirable. Many of these approaches have arisen out of frustrations with conventional financial support mechanisms as well as the perceived shortcomings of the current drug development system. The numbers I’ve posted above make clear that spending by pharma, biotech, and the government vastly dwarf the other players at paying for research and development work. However, the other organizations that invest their time, energy, and money are filling in gaps and enabling approaches (especially on the translational side) that might have not been undertaken otherwise. Financial resources have a way of waxing and waning as the political winds blow in different directions. I strongly favor having a diversity of financial resources because it is impossible to predict where the next breakthrough will come from. How the balance shifts among the various U.S. funding sources, both for profit and nonprofit, will depend on the overall health of the economy, the strength of the VC sector, and the generosity of philanthropists large and small to the nation’s disease-focused charities and other organizations.
